Regulatory Oversight and Compliance

Pharmaceutical training programs are enhanced by integrating ICH quality risk management considerations.

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post.

The treatment for ATTR in certain adults with polyneuropathy had just been recommended for approval across the European Union and had gained regulatory approval in the United States at the end of 2023.

A new laboratory will be built in Oss, while additional capacity and capabilities will be added to the Bioanalytical Center of Excellence in Assen.

Vyalev, also known as Produodopa, has been approved in 35 countries, with more than 4200 patients having started treatment.

This article presents an overview of FDA’s recent update to the guidance document, Control of Nitrosamine Impurities in Human Drugs.

Pfizer’s bivalent RSV prefusion F vaccine has been approved to prevent lower respiratory tract disease resulting from the respiratory syncytial virus in adults 18 to 59 years of age who are at increased risk.

As many as 30% of people living with severe hemophilia A develop inhibitors such as TFPI, which can be produced by the body’s immune response to clotting factors in replacement therapy, often limiting the effectiveness of that therapy.

Wainzua had been previously approved for use by patients with ATTRv-PN in the United States, under the brand name Wainua.

The agency’s CHMP recommended drugs to treatments for hemophilia, two biosimilars for psoriasis, and two flu vaccines at their October meeting.

The agency has reconfirmed its recommendation to not give positive opinion to Translarna (ataluren) after a reexamination of the available data.

The draft joint network strategy for the EU until 2028 will be open for public consultation until Nov. 30, 2024.

Zaim Gashi, area sales manager of Steriline, discussed the evolution of aseptic processing equipment in line with current regulatory requirements at CPHI Milan.

Regulators, EMA and HMA, have published principles and recommendations on the use of LLMs, which are being increasingly used for daily tasks.

The new mechanism of action targets cholinergic receptors instead of the standard dopamine receptors.

The agency is recommending that the market authorization for Oxbryta (voxelotor) be suspended so that emerging data may be reviewed.

Two drugs, Miplyffa (arimoclomol) and Aqneursa (levacetylleucine), have been approved by FDA to treat neurological symptoms associated with Niemann-Pick disease, type C in adults and children.

FDA’s Darby Kozak provided commentary on the anniversary of the Drug Price Competition and Patent Term Restoration Act of 1984.

The approval of EBGLYSS (lebrikizumab-lbkz) by FDA puts a new biologic treatment for moderate-to-severe atopic dermatitis on the market for patients whose symptoms are not well controlled with topical treatments.

Approvals were recommended for treatments of ovarian cancer, lung cancer, bleeding disorders, respiratory disease, and more.

The company said this new commitment to its facility in Germany goes hand in hand with its recently announced ReciPredict, an initiative intended to streamline the product development cycle.

Bio/pharma companies should be approaching their decarbonization journey as a strategic business move for the future as industry stakeholders are placing greater value on sustainability.

What impact will the AI Act have on pharma and digital medicine?

Phase III trial results showed that Imfinzi (durvalumab in combination with chemotherapy reduced the risk of recurrence, progression, or death by 32% in patients with resectable non-small cell lung cancer before and after surgery.

PiaSky (crovalimab) has previously been approved in countries such as the United States and Japan for monthly, subcutaneous use that may be self-administered with adequate training.

Opaganib was given orphan drug designation for the treatment of neuroblastoma in children, which is rare, but the most common infancy malignancy.

In a 24-week study of patients with PAH, six-minute walking distance improved by an average of 40.8 meters, and risk of death or clinical worsening was reduced by 82%.

The combination of Opdivo plus Yervoy is already established as a second-line treatment for patients with advanced hepatocellular carcinoma.

The International Coalition of Medicines Regulatory Authorities’ new Collaborative Hybrid Inspection Pilot program allows a CDMO to gain approval from multiple countries simultaneously, as opposed to separately phased inspections from different corners of the globe.

This Behind the Headlines news roundup panel discussion covers news items such as the resurgence of mpox in Africa, “medical tourism” in India, Eli Lilly’s new Boston CGT facilities, and contract development and manufacturing organization (CDMO) projected growth rates for the 2023–2029 period, and the reasons behind them.